International Drug Development Institute (Belgium)

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Title DOI
https://doi.org/10.1177/0962280207081864 Individual- and trial-level surrogacy in colorectal cancer
https://doi.org/10.1046/j.1468-2982.2000.00069.x Comparison of Rizatriptan 10 Mg vs. Zolmitriptan 2.5 Mg in the Acute Treatment of Migraine
https://doi.org/10.1017/s0266462314000300 VALIDATION OF SURROGATE ENDPOINTS IN ADVANCED SOLID TUMORS: SYSTEMATIC REVIEW OF STATISTICAL METHODS, RESULTS, AND IMPLICATIONS FOR POLICY MAKERS
https://doi.org/10.1093/annonc/mdp011 Radiological tumor size decrease at week 6 is a potent predictor of outcome in chemorefractory metastatic colorectal cancer treated with cetuximab (BOND trial)
https://doi.org/10.1182/blood-2011-02-337725 Individual patient data meta-analysis of randomized trials evaluating IL-2 monotherapy as remission maintenance therapy in acute myeloid leukemia
https://doi.org/10.1093/annonc/mdv562 Statistical controversies in clinical research: end points other than overall survival are vital for regulatory approval of anticancer agents
https://doi.org/10.1200/jco.2007.12.1368 End Points in Advanced Colon Cancer Clinical Trials: A Review and Proposal
https://doi.org/10.1158/1078-0432.ccr-14-2993 PTEN Loss Is Associated with Worse Outcome in HER2 -Amplified Breast Cancer Patients but Is Not Associated with Trastuzumab Resistance
https://doi.org/10.1093/jnci/djx179 Understanding and Communicating Measures of Treatment Effect on Survival: Can We Do Better?
https://doi.org/10.1007/s00280-013-2262-2 Safety and efficacy of neratinib in combination with weekly paclitaxel and trastuzumab in women with metastatic HER2-positive breast cancer: an NSABP Foundation Research Program phase I study
https://doi.org/10.3816/cbc.2010.n.011 A Phase II Neoadjuvant Trial of Sequential Nanoparticle Albumin-Bound Paclitaxel Followed by 5-Fluorouracil/Epirubicin/Cyclophosphamide in Locally Advanced Breast Cancer
https://doi.org/10.1200/jco.2011.38.6359 Overall Survival: Patient Outcome, Therapeutic Objective, Clinical Trial End Point, or Public Health Measure?
https://doi.org/10.3132/dvdr.2007.064 The European Perspective on Diabetes Prevention: development and Implementation of A European Guideline and training standards for diabetes prevention (IMAGE)
https://doi.org/10.1093/annonc/mdw098 Long-term outcomes after adjuvant treatment of sequential versus combination docetaxel with doxorubicin and cyclophosphamide in node-positive breast cancer: BCIRG-005 randomized trial
https://doi.org/10.1186/bcr3179 Prognostic and predictive value of TP53mutations in node-positive breast cancer patients treated with anthracycline- or anthracycline/taxane-based adjuvant therapy: results from the BIG 02-98 phase III trial
https://doi.org/10.1016/j.breast.2014.02.009 The impact of educational materials on compliance and persistence rates with adjuvant aromatase inhibitor treatment: First-year results from the Compliance of ARomatase Inhibitors AssessmenT In Daily practice through Educational approach (CARIATIDE) study
https://doi.org/10.1200/jco.19.03114 Event-Free Survival, a Prostate-Specific Antigen–Based Composite End Point, Is Not a Surrogate for Overall Survival in Men With Localized Prostate Cancer Treated With Radiation
https://doi.org/10.1191/0962280204sm362ra A perspective on surrogate endpoints in controlled clinical trials
https://doi.org/10.1177/1740774506070807 Impact of on-site initiation visits on patient recruitment and data quality in a randomized trial of adjuvant chemotherapy for breast cancer
https://doi.org/10.1136/bmjopen-2017-017075 Study protocol for THINK: a multinational open-label phase I study to assess the safety and clinical activity of multiple administrations of NKR-2 in patients with different metastatic tumour types
https://doi.org/10.1016/j.ophtha.2014.07.043 Stereotactic Radiotherapy for Neovascular Age-Related Macular Degeneration
https://doi.org/10.1186/s13058-019-1196-y NSABP FB-7: a phase II randomized neoadjuvant trial with paclitaxel + trastuzumab and/or neratinib followed by chemotherapy and postoperative trastuzumab in HER2+ breast cancer
https://doi.org/10.1093/annonc/mdv259 St Gallen International Expert Consensus on the primary therapy of early breast cancer: an invaluable tool for physicians and scientists
https://doi.org/10.1080/0094965031000062177 Simplified hierarchical linear models for the evaluation of surrogate endpoints
https://doi.org/10.1038/bjc.2011.235 Designing phase II trials in cancer: a systematic review and guidance
https://doi.org/10.1002/sim.6294 Linear mixed‐effects models for central statistical monitoring of multicenter clinical trials
https://doi.org/10.1200/jco.2010.34.0380 Progression-Free Survival Ratio As End Point for Phase II Trials in Advanced Solid Tumors
https://doi.org/10.3233/jad-150429 Protein Kinase Activity Decreases with Higher Braak Stages of Alzheimer’s Disease Pathology
https://doi.org/10.1016/s1470-2045(21)00077-2 The International Collaboration for Research methods Development in Oncology (CReDO) workshops: shaping the future of global oncology research
https://doi.org/10.3324/haematol.2010.039131 Leukemia-free survival as a surrogate end point for overall survival in the evaluation of maintenance therapy for patients with acute myeloid leukemia in complete remission
https://doi.org/10.1007/s40265-017-0728-y Progression-Free Survival as a Surrogate for Overall Survival in Clinical Trials of Targeted Therapy in Advanced Solid Tumors
https://doi.org/10.1016/j.ejca.2015.03.018 Final 10-year results of the Breast International Group 2–98 phase III trial and the role of Ki67 in predicting benefit of adjuvant docetaxel in patients with oestrogen receptor positive breast cancer
https://doi.org/10.1200/jco.2011.38.4206 Survival Is Not a Good Outcome for Randomized Trials With Effective Subsequent Therapies
https://doi.org/10.1634/theoncologist.2011-0212 Correlation Between Quantitative HER-2 Protein Expression and Risk for Brain Metastases in HER-2+ Advanced Breast Cancer Patients Receiving Trastuzumab-Containing Therapy
https://doi.org/10.1111/jch.12840 High Blood Pressure 2016: Why Prevention and Control Are Urgent and Important. The World Hypertension League, International Society of Hypertension, World Stroke Organization, International Diabetes Foundation, International Council of Cardiovascular Prevention and Rehabilitation, International Society of Nephrology
https://doi.org/10.1001/jamaoncol.2018.6012 Assessment ofERBB2/HER2Status inHER2-Equivocal Breast Cancers by FISH and 2013/2014 ASCO-CAP Guidelines
https://doi.org/10.2217/fon-2019-0201 Devimistat in Combination with High Dose Cytarabine and Mitoxantrone Compared with High Dose Cytarabine and Mitoxantrone in Older Patients with Relapsed/Refractory Acute Myeloid Leukemia: ARMADA 2000 Phase III Study
https://doi.org/10.1016/j.csda.2011.03.014 Comparative assessment of trial-level surrogacy measures for candidate time-to-event surrogate endpoints in clinical trials
https://doi.org/10.1186/s13058-019-1240-y Correction to: NSABP FB-7: a phase II randomized neoadjuvant trial with paclitaxel + trastuzumab and/or neratinib followed by chemotherapy and postoperative trastuzumab in HER2+ breast cancer
https://doi.org/10.1038/s41375-020-0733-0 ATIR101 administered after T-cell-depleted haploidentical HSCT reduces NRM and improves overall survival in acute leukemia
https://doi.org/10.2217/fon.14.179 Does patient education work in breast cancer? Final results from the global CARIATIDE study
https://doi.org/10.1038/bjc.2015.55 Assessing the benefit–risk of new treatments using generalised pairwise comparisons: the case of erlotinib in pancreatic cancer
https://doi.org/10.1016/j.cct.2020.106189 Infectious diseases epidemiology, quantitative methodology, and clinical research in the midst of the COVID-19 pandemic: Perspective from a European country
https://doi.org/10.1016/j.ejca.2023.02.002 Overall survival with neratinib after trastuzumab-based adjuvant therapy in HER2-positive breast cancer (ExteNET): A randomised, double-blind, placebo-controlled, phase 3 trial
https://doi.org/10.1016/j.ejca.2014.08.017 Association of adverse events and survival in colorectal cancer patients treated with adjuvant 5-fluorouracil and leucovorin: Is efficacy an impact of toxicity?
https://doi.org/10.1093/annonc/mdq406 Neoadjuvant capecitabine and docetaxel (plus trastuzumab): an effective non-anthracycline-based chemotherapy regimen for patients with locally advanced breast cancer
https://doi.org/10.1186/s13058-019-1115-2 PIK3CA alterations and benefit with neratinib: analysis from the randomized, double-blind, placebo-controlled, phase III ExteNET trial
https://doi.org/10.3747/co.v18is2.941 Progression-Free Survival as a Primary Endpoint in Clinical Trials of Metastatic Colorectal Cancer
https://doi.org/10.1177/009286150003400207 Randomized Designs for Early Trials of New Cancer Treatments—An Overview
https://doi.org/10.1182/blood-2019-123219 A Phase 1 Study of ALX148, a CD47 Blocker, in Combination with Rituximab in Patients with Non-Hodgkin Lymphoma
https://doi.org/10.1016/j.ygyno.2019.11.034 A randomized phase III trial in patients with recurrent platinum sensitive ovarian cancer comparing efficacy and safety of paclitaxel micellar and Cremophor EL-paclitaxel
https://doi.org/10.1182/blood-2020-135941 ALX148, a CD47 Blocker, in Combination with Rituximab in Patients with Non-Hodgkin Lymphoma
https://doi.org/10.4103/2229-3485.154016 Common pitfalls in statistical analysis: "P" values, statistical significance and confidence intervals
https://doi.org/10.1158/1078-0432.ccr-20-1831 Neratinib-Plus-Cetuximab in Quadruple-WT ( KRAS, NRAS, BRAF, PIK3CA ) Metastatic Colorectal Cancer Resistant to Cetuximab or Panitumumab: NSABP FC-7, A Phase Ib Study
https://doi.org/10.1111/biom.12483 An Information-Theoretic Approach for the Evaluation of Surrogate Endpoints Based on Causal Inference
https://doi.org/10.1007/s00415-016-8051-1 Normalisation of brain spectroscopy findings in Niemann–Pick disease type C patients treated with miglustat
https://doi.org/10.1016/j.jtho.2019.05.030 Chronological Trends in Progression-Free, Overall, and Post-Progression Survival in First-Line Therapy for Advanced NSCLC
https://doi.org/10.1093/annonc/mdp012 FOLFOX in patients with metastatic colorectal cancer and high alkaline phosphatase level: an exploratory cohort of the GERCOR OPTIMOX1 study
https://doi.org/10.1177/1740774519862564 Detection of atypical data in multicenter clinical trials using unsupervised statistical monitoring
https://doi.org/10.1080/10428194.2022.2043304 Momelotinib reduces transfusion requirements in patients with myelofibrosis
https://doi.org/10.1200/jco.2013.31.15_suppl.tps8121 A phase III comparative study of nivolumab (anti-PD-1; BMS-963558; ONO-4538) versus docetaxel in patients (pts) with previously treated advanced/metastatic nonsquamous non-small-cell lung cancer (NSCLC).
https://doi.org/10.1177/1740774517716158 The impact of data errors on the outcome of randomized clinical trials
https://doi.org/10.1200/jco.2020.38.15_suppl.3056 A phase I study of ALX148, a CD47 blocker, in combination with standard anticancer antibodies and chemotherapy regimens in patients with advanced malignancy.
https://doi.org/10.1177/0962280217718582 A Poisson approach to the validation of failure time surrogate endpoints in individual patient data meta-analyses
https://doi.org/10.1111/biom.12583 Estimation of Diagnostic Accuracy of a Combination of Continuous Biomarkers Allowing for Conditional Dependence Between the Biomarkers and the Imperfect Reference-Test
https://doi.org/10.1158/1078-0432.ccr-21-1762 Genetic Polymorphisms and Correlation with Treatment-Induced Cardiotoxicity and Prognosis in Patients with Breast Cancer
https://doi.org/10.1200/jco.2022.40.16_suppl.4023 Phase 3, multicenter, randomized study of CPI-613 with modified FOLFIRINOX (mFFX) versus FOLFIRINOX (FFX) as first-line therapy for patients with metastatic adenocarcinoma of the pancreas (AVENGER500).
https://doi.org/10.1200/jco.2012.30.15_suppl.611 A phase I dose-escalation study evaluating weekly paclitaxel with neratinib and trastuzumab in women with metastatic HER2-positive breast cancer, NSABP FB-8.
https://doi.org/10.1200/jco.2017.35.15_suppl.1002 A phase II trial of the CDK4/6 inhibitor palbociclib (P) as single agent or in combination with the same endocrine therapy (ET) received prior to disease progression, in patients (pts) with hormone receptor positive (HR+) HER2 negative (HER2−) metastatic breast cancer (mBC) (TREnd trial).
https://doi.org/10.1007/s43441-021-00341-5 Detection of Fraud in a Clinical Trial Using Unsupervised Statistical Monitoring
https://doi.org/10.1016/j.annonc.2023.11.017 Validation of metastasis-free survival as a surrogate endpoint for overall survival in localized prostate cancer in the era of docetaxel for castration-resistant prostate cancer
https://doi.org/10.1001/jamanetworkopen.2019.11750 Evaluation of Continuous Tumor-Size–Based End Points as Surrogates for Overall Survival in Randomized Clinical Trials in Metastatic Colorectal Cancer
https://doi.org/10.1177/17407745231206465 Design of a clinical trial using generalized pairwise comparisons to test a less intensive treatment regimen
https://doi.org/10.1158/1538-7445.sabcs14-s6-01 Abstract S6-01: Phase 3, randomized, double-blind, placebo-controlled multicenter trial of daily everolimus plus weekly trastuzumab and paclitaxel as first-line therapy in women with HER2+ advanced breast cancer: BOLERO-1
https://doi.org/10.1016/j.clbc.2016.07.008 Epirubicin With Cyclophosphamide Followed by Docetaxel With Trastuzumab and Bevacizumab as Neoadjuvant Therapy for HER2-Positive Locally Advanced Breast Cancer or as Adjuvant Therapy for HER2-Positive Pathologic Stage III Breast Cancer: A Phase II Trial of the NSABP Foundation Research Group, FB-5
https://doi.org/10.1080/0284186x.2020.1871067 Rationale and design of REGINA, a phase II trial of neoadjuvant regorafenib, nivolumab, and short-course radiotherapy in stage II and III rectal cancer
https://doi.org/10.1007/s13300-021-01030-0 Impact of Age on the Effectiveness and Safety of Insulin Glargine 300 U/mL: Results from the REALI European Pooled Data Analysis
https://doi.org/10.1016/j.radonc.2021.04.006 Independent external validation using the EORTC HNCG-ROG 1219 DAHANCA trial data of NTCP models for acute oral mucositis
https://doi.org/10.1016/j.adro.2024.101674 Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis
https://doi.org/10.1002/pst.2156 Operational characteristics of generalized pairwise comparisons for hierarchically ordered endpoints
https://doi.org/10.1136/jitc-2021-sitc2021.498 498 Evorpacept (ALX148), a CD47 myeloid checkpoint inhibitor, in patients with head and neck squamous cell carcinoma (HNSCC) and with gastric/gastroesophageal cancer (GC); ASPEN-01
https://doi.org/10.1002/bimj.201900354 A new measure of treatment effect in clinical trials involving competing risks based on generalized pairwise comparisons
https://doi.org/10.1093/jnci/djad152 Time to deterioration of patient-reported outcomes as a surrogate of overall survival: a meta-analysis
https://doi.org/10.1002/sim.8788 Net benefit in the presence of correlated prioritized outcomes using generalized pairwise comparisons: A simulation study
https://doi.org/10.1002/pst.2165 Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long‐term clinical endpoint
https://doi.org/10.1093/jnci/djab218 Clinical Trial Endpoints in Metastatic Cancer: Using Individual Participant Data to Inform Future Trials Methodology
https://doi.org/10.1200/jco.23.01196 Early Efficacy End Points in Neoadjuvant Rectal Cancer Trials: Surrogacy Revisited
https://doi.org/10.1007/s43441-024-00613-w Does Central Statistical Monitoring Improve Data Quality? An Analysis of 1,111 Sites in 159 Clinical Trials
https://doi.org/10.1186/s13058-024-01823-8 NSABP FB-10: a phase Ib/II trial evaluating ado-trastuzumab emtansine (T-DM1) with neratinib in women with metastatic HER2-positive breast cancer
https://doi.org/10.1200/jco.24.00539 Clinical Study Report and Individual Participant Data Transparency for US Food and Drug Administration–Approved Anticancer Drugs: A Call for Systematic Data Availability
https://doi.org/10.1007/s43441-022-00470-5 Does Central Monitoring Lead to Higher Quality? An Analysis of Key Risk Indicator Outcomes
https://doi.org/10.1111/epi.13410 Disruption, but not overexpression of urate oxidase alters susceptibility to pentylenetetrazole‐ and pilocarpine‐induced seizures in mice
https://doi.org/10.1080/00015458.2018.1470276 Impact of a prehospital discrimination between trauma patients with or without early acute coagulopathy of trauma and the need for damage control resuscitation: rationale and design of a multicenter randomized phase II trial
https://doi.org/10.1053/j.sodo.2025.06.002 Comparing prescribed and achieved treatment outcomes in digitally planned orthodontic treatment: statistical approaches
https://doi.org/10.1002/pst.2271 On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes
https://doi.org/10.1186/s13023-023-02943-8 Generalized pairwise comparisons of prioritized outcomes are a powerful and patient-centric analysis of multi-domain scores
https://doi.org/10.1016/j.esmogo.2024.100067 Efficacy and safety of short-course radiotherapy versus total neoadjuvant therapy in older rectal cancer patients: a randomised pragmatic trial (SHAPERS)
https://doi.org/10.1016/j.eclinm.2025.103465 Use of surrogate endpoints in health technology assessment and reimbursement of treatments for the management of chronic kidney disease
https://doi.org/10.1200/jco.2023.41.16_suppl.5073 The relationship between a priori defined prognostic risk groups and and overall survival (OS) in men with metastatic hormone sensitive prostate cancer (mHSPC).
https://doi.org/10.1016/j.jclinepi.2024.111340 Restricted Net Treatment Benefit in oncology